Curtin François, Heritier Stephane
a Division of Clinical Pharmacology and Toxicology , University of Geneva , Geneva , Switzerland.
b Research Center for Statistics, Geneva School of Economics and Management , University of Geneva , Geneva , Switzerland.
Expert Rev Clin Pharmacol. 2017 Jul;10(7):727-736. doi: 10.1080/17512433.2017.1321985. Epub 2017 Apr 28.
Clinical development of new drugs is a long and costly process. There is a need to find solutions which can improve and shorten this process. By introducing flexibility in to the design of clinical trials, adaptive design contributes to this improvement and allows to reach drug development decisions in a quicker way. Areas covered: We review the main methodological approaches to adaptive trial design, introducing key statistical concepts. For each phase of the clinical development, different uses and implementations of adaptive trial (AD) design are presented and examples of recent clinical trials are given. The guidance documents issued by the US and European regulatory authorities are also presented. Expert commentary: Despite inevitable challenges, prospects of this rapidly evolving approach to drug development are important. Controlling the risk of type 1 error and the potential operational risks which may be associated with adaptive trial strategy is paramount in late phase studies. However, with new methodological work, these risks are now well controlled and adaptive trial design will certainly shape the future of drug development.
新药的临床开发是一个漫长且成本高昂的过程。需要找到能够改进和缩短这一过程的解决方案。通过在临床试验设计中引入灵活性，适应性设计有助于实现这一改进，并能更快地做出药物开发决策。涵盖领域：我们回顾了适应性试验设计的主要方法学途径，介绍了关键的统计概念。针对临床开发的每个阶段，阐述了适应性试验（AD）设计的不同用途和实施方式，并给出了近期临床试验的实例。还介绍了美国和欧洲监管机构发布的指导文件。专家评论：尽管存在不可避免的挑战，但这种快速发展的药物开发方法的前景十分重要。在后期研究中，控制I类错误风险以及可能与适应性试验策略相关的潜在操作风险至关重要。然而，随着新的方法学工作的开展，这些风险现已得到很好的控制，适应性试验设计必将塑造药物开发的未来。
